Deflazacort versus prednisone treatment for Duchenne muscular dystrophy: a meta-analysis of disease progression rates in recent multicenter clinical trials

McDonald, Cm; Sajeev, G; Yao, Z; McDonnell, E; Elfring, G; Souza, M; Peltz, Sw; Darras, Bt; Shieh, Pb; Cox, Da; Landry, J; Signorovitch, J; Campbell, C; Torricelli, Re; Finkel, Rs; Flanigan, Km; Goemans, N; Heydemann, P; Kaminska, A; Kirschner, J; Muntoni, F; Osorio, An; Schara, U; Sejersen, T; Sweeney, Hl; Topaloglu, H; Tulinius, M; Vilchez, Jj; Voit, T; Wong, B; Alfano, Ln; Eagle, M; James, Mk; Lowes, L; Mayhew, A; Mazzone, Es; Nelson, L; Rose, Kj; Abdel-Hamid, Hz; Apkon, Sd; Barohn, Rj; Bertini, E; Bloetzer, C; de Vaud LC,; Butterfield, Rj; Chabrol, B; Chae, Jh; Jongno-Gu, Dr; Comi, Gp; Dastgir, J; Desguerre, I; Escobar, Rg; Finanger, E; Guglieri, M; Hughes, I; Iannaccone, St; Jones, Kj; Karachunski, P; Kudr, M; Lotze, T; Mah, Jk; Mathews, K; Nevo, Y; Parsons, J; Péréon, Y; de Queiroz Campos Araujo AP,; Renfroe, Jb; de Mbd, R; Ryan, M; Selby, K; Tennekoon, G; Vita, G; Abdel-Hamid, H; Apkon, S; Barohn, R; Belousova, E; Bertini, E; Brandsema, J; Bruno, C; Burnette, W; Butterfield, R; Byrne, B; Campbell, C; Carlo, J; Chae, Jh; Chandratre, S; Comi, G; Connolly, A; De Groot, I; Deconinck, N; Dooley, J; Dubrovsky, A; Durigneux, J; Finanger, E; Finkel, R; Frank, Lm; Goemans, N; Harper, A; Hattori, A; Herguner, O; Iannaccone, S; Janas, J; Jong, Yj; Kirschner, J; Komaki, H; Kuntz, N; Lee, Wt; Leung, E; Mah, J; Mathews, K; Cm, M; Mercuri, E; McMillan, H; Mueller-Felber, W; Lopez de Munain, A; Nakamura, A; Niks, E; Ogata, K; Pascual, S; Pegoraro, E; Pereon, Y; Renfroe, B; Sanka, Rb; Schallner, J; Schara, U; Selby, K; Sendra, Ii; Servais, L; Smith, E; Sparks, S; Topaloglu, H; Victor, R; Vilchez, Jj; Wicklund, M; Wilichoswki, E; Wong, B.

doi:10.1002/mus.26736

We characterized disease progression over 48 weeks among boys receiving deflazacort versus prednisone/prednisolone placebo arm treatment in two recent Duchenne muscular dystrophy (DMD) clinical trials. Ambulatory boys with DMD receiving placebo in the Phase 3 ataluren (n=115) and tadalafil (n=116) trials were included. The trials required ≥ 6 months of prior corticosteroid use and stable baseline dosing. Associations between corticosteroid and 48-week changes in ambulatory function were estimated using mixed models. Adjusted differences between corticosteroid groups were pooled in a meta-analysis. In the meta-analysis, deflazacort-treated patients versus prednisone/prednisolone-treated patients experienced, on average, lower declines of 28.3 meters on six-minute walk distance [95% confidence interval: (5.7, 50.9)], 2.9 seconds on rise from supine, [(0.9, 4.9)], 2.3 seconds on 4 stair climb [(0.5, 4.1)], and 2.9 points [(0. 1, 5.8)] on the North Star Ambulatory Assessment linearized score. Deflazacort-treated patients experienced significantly lower functional decline over 48 weeks.

Titolo:	Deflazacort versus prednisone treatment for Duchenne muscular dystrophy: a meta-analysis of disease progression rates in recent multicenter clinical trials
Autori:	McDonald CM Sajeev G Yao Z McDonnell E Elfring G Souza M Peltz SW Darras BT Shieh PB Cox DA Landry J Signorovitch J Campbell C Torricelli RE Finkel RS Flanigan KM Goemans N Heydemann P Kaminska A Kirschner J Muntoni F Osorio AN Schara U Sejersen T Sweeney HL Topaloglu H Tulinius M Vilchez JJ Voit T Wong B Alfano LN Eagle M James MK Lowes L Mayhew A Mazzone ES Nelson L Rose KJ Abdel-Hamid HZ Apkon SD Barohn RJ Bertini E Bloetzer C de Vaud LC Butterfield RJ Chabrol B Chae JH Jongno-Gu DR Comi GP Dastgir J Desguerre I Escobar RG Finanger E Guglieri M Hughes I Iannaccone ST Jones KJ Karachunski P Kudr M Lotze T Mah JK Mathews K Nevo Y Parsons J Péréon Y de Queiroz Campos Araujo AP Renfroe JB de Mbd R Ryan M Selby K Tennekoon G VITA, Giuseppe Abdel-Hamid H Apkon S Barohn R Belousova E Bertini E Brandsema J Bruno C Burnette W Butterfield R Byrne B Campbell C Carlo J Chae JH Chandratre S Comi G Connolly A De Groot I Deconinck N Dooley J Dubrovsky A Durigneux J Finanger E Finkel R Frank LM Goemans N Harper A Hattori A Herguner O Iannaccone S Janas J Jong YJ Kirschner J Komaki H Kuntz N Lee WT Leung E Mah J Mathews K Cm M Mercuri E McMillan H Mueller-Felber W Lopez de Munain A Nakamura A Niks E Ogata K Pascual S Pegoraro E Pereon Y Renfroe B Sanka RB Schallner J Schara U Selby K Sendra II Servais L Smith E Sparks S Topaloglu H Victor R Vilchez JJ Wicklund M Wilichoswki E Wong B. mostra contributor esterni nascondi contributor esterni
Data di pubblicazione:	2020
Rivista:	MUSCLE & NERVE
Abstract:	We characterized disease progression over 48 weeks among boys receiving deflazacort versus prednisone/prednisolone placebo arm treatment in two recent Duchenne muscular dystrophy (DMD) clinical trials. Ambulatory boys with DMD receiving placebo in the Phase 3 ataluren (n=115) and tadalafil (n=116) trials were included. The trials required ≥ 6 months of prior corticosteroid use and stable baseline dosing. Associations between corticosteroid and 48-week changes in ambulatory function were estimated using mixed models. Adjusted differences between corticosteroid groups were pooled in a meta-analysis. In the meta-analysis, deflazacort-treated patients versus prednisone/prednisolone-treated patients experienced, on average, lower declines of 28.3 meters on six-minute walk distance [95% confidence interval: (5.7, 50.9)], 2.9 seconds on rise from supine, [(0.9, 4.9)], 2.3 seconds on 4 stair climb [(0.5, 4.1)], and 2.9 points [(0. 1, 5.8)] on the North Star Ambulatory Assessment linearized score. Deflazacort-treated patients experienced significantly lower functional decline over 48 weeks.
Handle:	http://hdl.handle.net/11570/3145121
Appare nelle tipologie:	14.a.1 Articolo su rivista

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